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Generating drug-screenable primary human intestinal epithelium by gene-editing and transgenesis

Award Number: R43DK125155
ORGANIZATION: NATIONAL INSTITUTE OF DIABETES & DIGESTIVE & KIDNEY DISEASES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 04/01/2020
PERIOD OF PERFORMANCE END DATE: 09/30/2021

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Generating drug-screenable primary human intestinal epithelium by gene-editing and transgenesis - Project Summary: Maintaining healthy physiology of the human gut is a large focus of Pharma. Models to study drug/nutrient absorption, xenobiotics, toxicology, and preclinical efficacies are hampered by the lack of accurate, reproducible, and easy to use cell culture models to evaluate such topics. For these reasons, there is a strong need for better in vitro models that recapitulate disease states of the human gut, and better platforms for drug discovery and validation. To meet this need Altis Biosystems Inc., an early stage biotechnology company, will collaborate with scientists at the University of North Carolina at Chapel Hill to develop novel, rapidly screenable cells that can be generated on, and applied to, their gut stem cell-based technology called RepliGut™, which is a monolayer of human epithelium derived the small intestine or colon of organ donors, and contains all of the proliferative and differentiated cell types found in vivo. This Phase I study will 1) develop and optimize novel methods to for generating robust fluorescent protein based cell lines by transgenesis or CRISPR-gene editing, 2) create and validate two screenable cell lines to report key properties associated with gut health and disease - epithelial barrier function and inflammation, and 3) perform a proof-of-concept small scale compound screen to demonstrate utility and provide rationale for expanded development. These screenable cells will be developed with the eventual goal during Phase II of creating screenable cells and genetic disease model on RepliGut™ that can fit into high-throughput and high-content screening infrastructure. This collaboration between academia, Altis, and the NIH represents an ideal opportunity to further develop RepliGut™ for translation of this highly physiologic culture and screening platform into an underdeveloped marketplace.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2022 ( Subtotal = $0 )
2022	2020	ALTIS BIOSYSTEMS, INC.	6 DAVIS DR	DURHAM	NC	27709	DURHAM	USA	Diabetes, Digestive, and Kidney Diseases Extramural Research	000	1	4/28/2022	NEW	$0
														Subtotal = $0

Issue Date FY: 2020 ( Subtotal = $245,920 )
2020	2020	ALTIS BIOSYSTEMS, INC.	1289 N FORDHAM BLVD STE A234	CHAPEL HILL	NC	27514	ORANGE	USA	Diabetes, Digestive, and Kidney Diseases Extramural Research	000	1	3/30/2020	NEW	$245,920
														Subtotal = $245,920

Grand Total All Awards = $245,920

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Generating drug-screenable primary human intestinal epithelium by gene-editing and transgenesis

Award Number: R43DK125155

ORGANIZATION: NATIONAL INSTITUTE OF DIABETES & DIGESTIVE & KIDNEY DISEASES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 04/01/2020

PERIOD OF PERFORMANCE END DATE: 09/30/2021

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